Sarcoglycanopathies or LGMDR3-6 are rare autosomal recessive diseases affecting mainly skeletal and cardiac muscle. The onset occurs in childhood and the disease is progressive. Missense mutations, responsible for the majority of the reported cases, originate a folding-defective, albeit potentially functional sarcoglycan (SG), which is discarded by the cells' quality control system. This leads to the disruption of the SG-complex, a key player in assuring sarcolemma stability; indeed, its loss results in progressive muscle degeneration. In the recent years, with the intent to rescue the SG-complex, we exploited the use of protein folding correctors belonging to the CFTR modulators family. CFTR correctors, initially developed for cystic fibrosis, have been proved to be effective in rescuing different SG-mutants as well. The proof of concept was first established in heterologous cell models and then in myogenic cells from sarcoglycanopathy patients. Recently, in vivo validation of the CFTR corrector C17, our lead compound, has been accomplished in a novel mouse model of the disease, in which the hindlimbs express the R98H mutated form of the human α-SG. The outcome of a chronic treatment of five weeks of daily intraperitoneal injection of the small molecule was the rescue of the SG-complex at the sarcolemma, the amelioration of the dystrophic phenotype, the reduction of the creatine kinase plasma level, and, notably, the almost full recovery of the muscle force. In parallel, we evaluated the absorption, distribution, metabolism and elimination (ADME studies) of corrector C17, evidencing a reasonable drug-like profile, with an interesting longer persistency in skeletal muscle and heart, the target tissues in sarcoglycanopathies. Preliminary toxicological experiments have shown a good safety profile of the small molecule. Altogether, our data highlight corrector C17 as a good drug-candidate, complementary to gene-therapy, to be further developed for sarcoglycanopathies.